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First product for rare hepatic veno-occlusive disease launched

News, 27 May 2014

The first licensed product for the treatment of severe hepatic veno-occlusive disease (severe VOD or sVOD) in patients over one month of age undergoing haematopoietic stem cell transplantation (HSCT) therapy, Defitelio (defibrotide), has been launched.

Severe VOD, one of the most serious early complications in HSCT therapy, is associated with multi-organ failure and is fatal in over 80% of patients. HSCTs are performed with curative intent in patients with haematological malignancies, selected solid tumours and some non-malignant disorders, such as serious haemoglobinopathies.

“Severe VOD is a complex and unpredictable disease, and its impact on patients, physicians and resources is substantial. Early and effective intervention is crucial in saving lives and limiting the potentially significant burden of this disease, and physicians have been eagerly awaiting the commercial availability of Defitelio in Europe,” said Professor Mohamad Mohty, President-Elect of the EBMT and Professor of Haematology, Saint-Antoine Hospital and University Pierre & Marie Curie, Paris.

The efficacy of Defitelio to treat sVOD in HSCT patients is supported by data from a pivotal, multi-centre Phase 3 trial that evaluated Defitelio for the treatment of sVOD compared with a historical control group of patients who had received standard supportive care. In this trial, Defitelio was shown to provide a significant increase in survival rates for patients with sVOD in HSCT. The results demonstrated a 52% increase in survival at 100 days after transplantation for patients treated with Defitelio compared to patients in the historical control group (38.2% in the Defitelio group vs. 25.0% in the historical control group; p=0.0341). In the clinical trial, 23.5% of patients treated with Defitelio achieved complete response at 100 days after transplantation versus 9.4% of patients in the historical control group (p=0.013).

Treatment with Defitelio has generally been well tolerated in all age groups. In the phase 3 pivotal trial, the overall incidence of adverse events was similar in the Defitelio treatment group and in the control group.

In the EU, VOD is designated as a rare disease, affecting less than five in 10,000 people. Studies have reported a wide range of incidence rates for VOD. Generally, data indicate that approximately 14% of patients undergoing HSCT develop VOD.